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    Summary
    EudraCT Number:2019-001545-41
    Sponsor's Protocol Code Number:KO-MEN-001
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-05-26
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2019-001545-41
    A.3Full title of the trial
    A Phase 1/2 First in Human Study of the Menin-MLL(KMT2A) Inhibitor KO-539 in Patients with Relapsed or Refractory Acute Myeloid Leukemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    First in human clinical trial of the study drug KO-539 in patients with leukemia
    A.4.1Sponsor's protocol code numberKO-MEN-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04067336
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorKura Oncology, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportKura Oncology, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKura Oncology, Inc.
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address2 Seaport Lane, Suite 8A
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02110
    B.5.3.4CountryUnited States
    B.5.6E-mailmcyr@kuraoncology.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameziftomenib
    D.3.2Product code KO-539
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNziftomenib
    D.3.9.1CAS number 2134675-36-6
    D.3.9.2Current sponsor codeKO-539, C17060755-E
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameziftomenib
    D.3.2Product code KO-539
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNziftomenib
    D.3.9.1CAS number 2134675-36-6
    D.3.9.2Current sponsor codeKO-539, C17060755-E
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Relapsed and/or refractory Acute Myeloid Leukemia
    E.1.1.1Medical condition in easily understood language
    Relapsed and/or refractory leukemia
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10060558
    E.1.2Term Acute myeloid leukemia recurrent
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Phase 1 Part 1a: Dose-Escalation
    - To determine the MTD and/or the RP2D of ziftomenib in patients with R/R AML

    Phase 1 Part 1b: Dose-Validation/ Cohort Expansion
    - Determine the safety, tolerability and MBED of ziftomenib in biomarker-specific dosing cohorts, which have demonstrated early biological activity and have been determined to be safe as part of the dose-escalation phase

    Phase 2:
    - Assess evidence of ALA of ziftomenib in patients with NPM1-m R/R AML
    E.2.2Secondary objectives of the trial
    Phase 1 Part 1a: Dose-Escalation
    - To investigate the safety and tolerability of ziftomenib in patients with R/R AML
    - To characterize the PK of ziftomenib and metabolites after single oral (PO) dose administration and after multiple PO dose administrations.
    - Explore early evidence of ALA in patients with NPM1-m R/R AML

    Phase 1 Part 1b: Dose-Validation/ Cohort Expansion
    - Explore early evidence of ALA in patients with R/R AML

    Phase 2:
    - Assess evidence of clinical activity of ziftomenib in patients with NPM1-m R/R AML
    - Assess safety and tolerability of ziftomenib in patients with NPM1-m R/R AML
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Sub-study 1: Ziftomenib Effect on CYP3A4-mediated Metabolism - Phase 1 sub-study not applicable in EU
    • Title: SUB-STUDY TO EVALUATE THE EFFECT OF ZIFTOMENIB ON CYP3A4-MEDIATED METABOLISM OF MIDAZOLAM
    • Data and version: Version 8.0 dated 11May2023
    • Primary Objective:
    1. Determine the effect of ziftomenib on the PK of a single oral dose of midazolam in patients with R/R AML, excluding KMT2A-r and NPM1-m genetic subtypes

    Sub-study 2: CYP3A4 Inhibition Effect on Ziftomenib Exposure - Phase 1 sub-study not applicable in EU
    • Title: SUB-STUDY TO EVALUATE THE EFFECT OF CYP3A4 INHIBITION ON ZIFTOMENIB EXPOSURE
    • Data and version: Version 8.0 dated 11May2023
    • Primary Objective:
    1. Determine the effect of itraconazole on the PK of ziftomenib and ziftomenib metabolites in patients with R/R AML, excluding KMT2A-r and NPM1-m genetic subtypes
    • Secondary Objective:
    1. To evaluate the relationship between ziftomenib plasma concentrations and QTc intervals

    Sub-study 3: Ziftomenib in Acute Lymphoblastic Leukemia - Phase 1 sub-study with enrollment to be opened in UK and EU
    • Title: SUB-STUDY TO EVALUATE ZIFTOMENIB IN PATIENTS WITH RELAPSED OR REFRACTORY ACUTE LYMPHOBLASTIC LEUKEMIA
    • Data and version: Version 8.0 dated 11May2023
    • Primary Objective:
    1. Determine the safety, tolerability, and MBED/RP2D of ziftomenib in patients with KMT2A-r R/R ALL
    • Secondary Objective:
    1. Explore early evidence of clinical activity in patients with KMT2A-r R/R ALL
    E.3Principal inclusion criteria
    Key Inclusion Criteria:
    1. Relapsed/refractory (R/R) acute myeloid leukemia (AML) defined as those who have also failed or are not appropriate for any approved standard-of-care (SOC) therapies or hematopoietic stem cell transplant (HSCT)with reappearance of ≥ 5% blasts in the bone marrow (BM).
    2. ≥ 18 years of age.
    3. Eastern Cooperative Oncology Group performance status of ≤ 2, and a life expectancy of at least 2 months.
    4. Peripheral white blood cell counts ≤ 30,000/μL.
    E.4Principal exclusion criteria
    Key Exclusion Criteria
    1. Diagnosis of acute promyelocytic leukemia or chronic myelogenous leukemia in blast crisis.
    2. Donor lymphocyte infusion < 30 days prior to study entry.
    3. Clinically active central nervous system (CNS) leukemia.
    4. Has undergone HSCT and have not had adequate hematologic recovery (Recovery is defined as peripheral absolute neutrophil count (ANC) ≥ 1 × 10^9/L and platelets at least ≥ 50 × 10^9/L [but preferably ≥ 100 × 109/L] and signs of a cellular BM).
    5. Patients on immunosuppressive therapy post HSCT must be off all immunosuppression therapy within 2 weeks of Cycle 1 Day 1.
    6. ≥ Grade 2 active GvHD, moderate or severe limited chronic GvHD, or extensive chronic GvHD of any severity.
    7. Has received prior menin inhibitor
    8. Has received chemotherapy, immunotherapy, radiotherapy (unless if given for management of CNS leukemia), or any ancillary therapy that is considered to be investigational (ie, used for non-approved indications(s) and in the context of a research investigation) < 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug, whichever is shorter, to ensure that patient time without necessary AML therapy is appropriately limited.
    9. Requires treatment with concomitant drugs that are strong inhibitors or inducers of CYP3A4 with the exception of antibiotics, antifungals, and antivirals that are used as SOC to prevent or treat infections. Other such drugs that are considered absolutely essential by the Investigator for the care of the patient should be discussed on a case-by-case basis with the Kura Medical Monitor.
    10. Has an active uncontrolled acute or chronic systemic fungal, bacterial, viral, or other infection.
    11. Significant cardiovascular disease including unstable angina pectoris, uncontrolled hypertension or arrhythmia, history of cerebrovascular accident including transient ischemic attack within the past 6 months, congestive heart failure (New York Heart Association Class III or IV) related to primary cardiac disease, ischemic or severe valvular heart disease, or a myocardial infarction within 6 months prior to the first dose of study treatment.
    12. Mean corrected QT interval by Fredericia's formula > 480 ms on triplicate electrocardiograms.
    E.5 End points
    E.5.1Primary end point(s)
    Phase 1 Part a:
    - Maximum Tolerated Dose (MTD)
    - Recommended Phase 2 dose (RP2D)

    Phase 1 Part b:
    - Safety, tolerability, and efficacy at multiple timepoints during the study

    Phase 2:
    - Based on evaluation of CR/CRh
    E.5.1.1Timepoint(s) of evaluation of this end point
    At any time during the clinical trial. Disease assessment will be done when there is clinical evidence of response.
    E.5.2Secondary end point(s)
    Phase 1 Part a:
    - Safety/tolerability assessed at multiple timepoints during the study
    - From blood samples collected at specified timepoints during treatment
    - Plasma concentrations of ziftomenib and metabolite(s)
    - Plasma PK parameters (Cmax, Cmin, Tmax, AUC0-t, AUC0-8, CL/F, Vz/F, and t½)
    - Based on evaluation of (including but not limited to): a. Duration of CR/CRh, b. TI, c. CR/CRh MRD negativity, d. CRc (CR+ CRh + CRi), e. ORR (CR + CRh + CRi + MLFS), f. EFS, g. OS

    Phase 1 Part b:
    - Based on evaluation of (including but not limited to): a. Duration of CR/CRh, b. TI, c. CR/CRh MRD negativity, d. CRc e. EFS, f. OS

    Phase 2:
    - Based on evaluation of (including but not limited to): a. Duration of CR/CRh, b. TI, c. CR/CRh MRD negativity, d. CRc e. EFS, f. OS
    - Safety and tolerability assessed at multiple timepoints during the study
    E.5.2.1Timepoint(s) of evaluation of this end point
    At any time during the clinical trial. Disease assessment will be done when there is clinical evidence of response.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase I Part a: First in human - Dose escalation. Phase I Part b: Dose- Validation/ Cohort Expansion
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA34
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    United Kingdom
    United States
    Belgium
    France
    Germany
    Italy
    Poland
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last visit of the last subject (LVLS)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 118
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 118
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 236
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Other antineoplastic therapy according to investigator's decision. Subjects will be followed for safety until initiation of another anticancer treatment.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-08-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-09
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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